Cereno Scientific has reported positive topline results from its Phase I trial of CS014, a novel histone deacetylase (HDAC) inhibitor in development for idiopathic pulmonary fibrosis (IPF).
The Swedish biotech company said the results support advancing the program into Phase II clinical development, planned for the first half (H1) of 2026.
IPF is a chronic, progressive lung disease characterized by scarring of lung tissue, leading to respiratory failure. Current therapies are aimed at slowing disease progression.
Promising safety and tolerability results
The Phase I trial, conducted in Uppsala, Sweden, evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple ascending oral doses of CS014 in 48 healthy volunteers. The study included two parts: single ascending doses (SAD) in 30 participants and multiple ascending doses (MAD) administered over seven days in 18 participants.
As per Cereno, CS014 demonstrated a favorable safety and tolerability profile. There were no serious adverse events (SAEs), no early withdrawals, and no deaths. All treatment-related adverse events (AEs) were mild, transient, and fully resolved.
Notably, CS014 achieved blood levels at and above those predicted from non-clinical studies to have therapeutic potential in reversing pulmonary vascular remodeling and fibrosis. These processes are key pathological drivers in IPF and several other rare cardiovascular and pulmonary diseases.
“We are very pleased with the outcome of this study and the excellent collaboration with the CRO CTC,” said Rahul Agrawal, CMO and Head of R&D at Cereno Scientific. “CS014 was safe and well tolerated at exposure levels expected to impact pathological pulmonary vascular remodeling and reduction of fibrosis.”
A differentiated approach to IPF treatment
CS014’s mechanism of action as an HDAC inhibitor involves epigenetic modulation, which may provide a unique and differentiated treatment strategy for IPF. Currently, treatment options for IPF are limited and do not reverse fibrosis.
“CS014 is a cornerstone of our broader HDAC inhibitor portfolio, which we believe holds significant disease-modifying potential across a range of rare cardiovascular and pulmonary diseases,” said Sten R. Sörensen, CEO of Cereno Scientific. “These positive Phase I results, combined with strong non-clinical data, give us confidence as we advance into Phase II clinical development.”
Cereno plans to initiate a Phase II trial for CS014 in H1 2026. Full Phase I results will be submitted for publication in a peer-reviewed journal. The latest milestone follows Cereno’s earlier progress with its pulmonary arterial hypertension (PAH) drug candidate, which recently won FDA endorsement for a Phase IIb trial.
